title: Relationship between labile plasma iron, liver iron concentration and cardiac response in a deferasirox monotherapy trial.
abstract: The US04 trial was a multicenter, open-label, single arm trial of deferasirox monotherapy (30-40 mg/kg/day) for 18 months. Cardiac iron response was bimodal with improvements observed in patients with mild to moderate initial somatic iron stores; relationship of cardiac response to labile plasma iron is now presented. Labile plasma iron was measured at baseline, six months, and 12 months. In patients having a favorable cardiac response at 18 months, initial labile plasma iron was elevated in only 31% of patients at baseline and no patient at six or 12 months. Cardiac non-responders had elevated labile plasma iron in 50% of patients at baseline, 50% patients at six months, and 38% of patients at 12 months. Risk of abnormal labile plasma iron and cardiac response increased with initial liver iron concentration. Persistently increased labile plasma iron predicts cardiac non-response to deferasirox but labile plasma iron suppression does not guarantee favorable cardiac outcome. Study registered at www.clinicaltrials.gov (NCT00447694).
authors.affiliation: Children's Hospital of Los Angeles, Los Angeles CA 90027, USA. JWood@chla.usc.edu
authors.forename: John C
authors.initials: JC
authors.lastname: Wood
authors.forename: Tara
authors.initials: T
authors.lastname: Glynos
authors.forename: Alexis
authors.initials: A
authors.lastname: Thompson
authors.forename: Patricia
authors.initials: P
authors.lastname: Giardina
authors.forename: Paul
authors.initials: P
authors.lastname: Harmatz
authors.forename: Barinder P
authors.initials: BP
authors.lastname: Kang
authors.forename: Carole
authors.initials: C
authors.lastname: Paley
authors.forename: Thomas D
authors.initials: TD
authors.lastname: Coates
mesh_terms: D001565:Benzoates / Q000494:pharmacology / Q000627:therapeutic use; D000077588:Deferasirox; D005293:Ferritins / Q000097:blood; D006321:Heart / Q000187:drug effects*; D006801:Humans; D007501:Iron / Q000097:blood*; D007502:Iron Chelating Agents / Q000494:pharmacology / Q000627:therapeutic use; D019190:Iron Overload / Q000175:diagnosis / Q000188:drug therapy* / Q000378:metabolism; D008099:Liver / Q000378:metabolism*; D009206:Myocardium / Q000378:metabolism*; D011379:Prognosis; D016896:Treatment Outcome; D014230:Triazoles / Q000494:pharmacology / Q000627:therapeutic use; D017086:beta-Thalassemia / Q000175:diagnosis / Q000188:drug therapy / Q000378:metabolism
chemical_list: D001565:Benzoates; D007502:Iron Chelating Agents; D014230:Triazoles; D005293:Ferritins; D007501:Iron; D000077588:Deferasirox